Clinical Trials | Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination with Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients with Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

The University of Virginia Comprehensive Cancer Center seeks participants ages 18 and over with Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia that has adequate liver and renal function. The main purpose of this study is to: - investigate the potential risks and benefits of adding a targeted therapy called ziftomenib to intensive therapy (7+3) with cytarabine consolidation and seeing if the use of ziftomenib as maintenance therapy following consolidation is beneficial. - investigate the potential risks and benefits of adding a targeted therapy called ziftomenib to nonintensive therapy (ven/aza). Ziftomenib and placebo are referred to as the “study drug” in this research study. “Randomized” means you are assigned to your study treatment group at random or by chance, like pulling numbers from a hat. In the intensive cohort, you will be randomized to one of three study treatment groups: • Group 1 (33%): 7+3 in combination with ziftomenib during induction, ziftomenib+cytarabine consolidation, and ziftomenib maintenance • Group 2 (33%): 7+3 in combination with ziftomenib during induction, ziftomenib+cytarabine consolidation, and placebo maintenance • Group 3 (33%): 7+3 in combination with placebo during induction, placebo+cytarabine consolidation, and placebo maintenance In the nonintesnsive cohort, you will be randomized to one of two study treatment groups: • Group 1 (50%): ven/aza in combination with ziftomenib • Group 2 (50%): ven/aza in combination with placebo What we learn from this study may benefit future patients with your type of cancer. We may also learn information about your health as part of the research study. The site study team will keep you informed of any new information learned about your personal health while participating in the study. Taking part in the study may or may not improve your health. Your cancer may stay the same, get worse, or get better. The study drugs you receive may cause side effects and discomfort. Not all participants will stay on the study for the same amount of time. Your time on study will depend on how well you tolerate the study drug, how your cancer responds to the study treatment, and how you feel. We estimate that participants will be on the study treatment for around 2.5 years and that the total time on study will be about 3 years. You will be contacted by the study team for up to 3 years after Cycle 1 Day 1 to report any new symptoms. Additional information found here: Study Details | Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML | ClinicalTrials.gov For questions, contact: [email protected]